CA2576923A1 - Vecteur - Google Patents
Vecteur Download PDFInfo
- Publication number
- CA2576923A1 CA2576923A1 CA002576923A CA2576923A CA2576923A1 CA 2576923 A1 CA2576923 A1 CA 2576923A1 CA 002576923 A CA002576923 A CA 002576923A CA 2576923 A CA2576923 A CA 2576923A CA 2576923 A1 CA2576923 A1 CA 2576923A1
- Authority
- CA
- Canada
- Prior art keywords
- liposome
- sirna
- delivery vector
- vector according
- group
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Abandoned
Links
- 239000013598 vector Substances 0.000 title claims abstract description 132
- 239000002502 liposome Substances 0.000 claims abstract description 196
- 150000002632 lipids Chemical class 0.000 claims abstract description 131
- 238000012384 transportation and delivery Methods 0.000 claims abstract description 125
- 229920000642 polymer Polymers 0.000 claims abstract description 68
- 108020004459 Small interfering RNA Proteins 0.000 claims abstract description 18
- -1 cationic lipid Chemical class 0.000 claims description 80
- 239000003795 chemical substances by application Substances 0.000 claims description 76
- 238000000034 method Methods 0.000 claims description 72
- 239000000203 mixture Substances 0.000 claims description 49
- 238000010168 coupling process Methods 0.000 claims description 47
- 238000005859 coupling reaction Methods 0.000 claims description 47
- 230000008878 coupling Effects 0.000 claims description 42
- MWRBNPKJOOWZPW-CLFAGFIQSA-N dioleoyl phosphatidylethanolamine Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OCC(COP(O)(=O)OCCN)OC(=O)CCCCCCC\C=C/CCCCCCCC MWRBNPKJOOWZPW-CLFAGFIQSA-N 0.000 claims description 38
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 36
- 125000001183 hydrocarbyl group Chemical group 0.000 claims description 35
- 125000002091 cationic group Chemical group 0.000 claims description 34
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims description 32
- 201000010099 disease Diseases 0.000 claims description 31
- 210000000056 organ Anatomy 0.000 claims description 22
- 125000005647 linker group Chemical group 0.000 claims description 21
- 125000002344 aminooxy group Chemical group [H]N([H])O[*] 0.000 claims description 20
- 239000003446 ligand Substances 0.000 claims description 20
- GUBGYTABKSRVRQ-QKKXKWKRSA-N Lactose Natural products OC[C@H]1O[C@@H](O[C@H]2[C@H](O)[C@@H](O)C(O)O[C@@H]2CO)[C@H](O)[C@@H](O)[C@H]1O GUBGYTABKSRVRQ-QKKXKWKRSA-N 0.000 claims description 16
- 239000008194 pharmaceutical composition Substances 0.000 claims description 15
- GUBGYTABKSRVRQ-XLOQQCSPSA-N Alpha-Lactose Chemical compound O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@H]1O[C@@H]1[C@@H](CO)O[C@H](O)[C@H](O)[C@H]1O GUBGYTABKSRVRQ-XLOQQCSPSA-N 0.000 claims description 14
- 235000012000 cholesterol Nutrition 0.000 claims description 14
- 239000008101 lactose Substances 0.000 claims description 14
- 238000011282 treatment Methods 0.000 claims description 13
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 claims description 12
- 229930006000 Sucrose Natural products 0.000 claims description 12
- 239000005720 sucrose Substances 0.000 claims description 12
- 150000001720 carbohydrates Chemical class 0.000 claims description 11
- 239000002577 cryoprotective agent Substances 0.000 claims description 11
- 238000004519 manufacturing process Methods 0.000 claims description 11
- 238000002360 preparation method Methods 0.000 claims description 10
- 125000002723 alicyclic group Chemical group 0.000 claims description 9
- 235000014633 carbohydrates Nutrition 0.000 claims description 9
- 235000000346 sugar Nutrition 0.000 claims description 9
- HDTRYLNUVZCQOY-UHFFFAOYSA-N α-D-glucopyranosyl-α-D-glucopyranoside Natural products OC1C(O)C(O)C(CO)OC1OC1C(O)C(O)C(O)C(CO)O1 HDTRYLNUVZCQOY-UHFFFAOYSA-N 0.000 claims description 8
- HDTRYLNUVZCQOY-WSWWMNSNSA-N Trehalose Natural products O[C@@H]1[C@@H](O)[C@@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@@H](O)[C@@H](CO)O1 HDTRYLNUVZCQOY-WSWWMNSNSA-N 0.000 claims description 8
- HDTRYLNUVZCQOY-LIZSDCNHSA-N alpha,alpha-trehalose Chemical compound O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CO)O[C@@H]1O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 HDTRYLNUVZCQOY-LIZSDCNHSA-N 0.000 claims description 8
- 239000003085 diluting agent Substances 0.000 claims description 8
- 150000001299 aldehydes Chemical class 0.000 claims description 7
- 208000019423 liver disease Diseases 0.000 claims description 7
- 230000008569 process Effects 0.000 claims description 7
- 206010067125 Liver injury Diseases 0.000 claims description 6
- 231100000234 hepatic damage Toxicity 0.000 claims description 6
- 230000008818 liver damage Effects 0.000 claims description 6
- 150000008104 phosphatidylethanolamines Chemical class 0.000 claims description 6
- DBTMGCOVALSLOR-UHFFFAOYSA-N 32-alpha-galactosyl-3-alpha-galactosyl-galactose Natural products OC1C(O)C(O)C(CO)OC1OC1C(O)C(OC2C(C(CO)OC(O)C2O)O)OC(CO)C1O DBTMGCOVALSLOR-UHFFFAOYSA-N 0.000 claims description 5
- RXVWSYJTUUKTEA-UHFFFAOYSA-N D-maltotriose Natural products OC1C(O)C(OC(C(O)CO)C(O)C(O)C=O)OC(CO)C1OC1C(O)C(O)C(O)C(CO)O1 RXVWSYJTUUKTEA-UHFFFAOYSA-N 0.000 claims description 5
- WQZGKKKJIJFFOK-QTVWNMPRSA-N D-mannopyranose Chemical compound OC[C@H]1OC(O)[C@@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-QTVWNMPRSA-N 0.000 claims description 5
- 229930091371 Fructose Natural products 0.000 claims description 5
- RFSUNEUAIZKAJO-ARQDHWQXSA-N Fructose Chemical compound OC[C@H]1O[C@](O)(CO)[C@@H](O)[C@@H]1O RFSUNEUAIZKAJO-ARQDHWQXSA-N 0.000 claims description 5
- 239000005715 Fructose Substances 0.000 claims description 5
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 claims description 5
- 238000004108 freeze drying Methods 0.000 claims description 5
- 239000008103 glucose Substances 0.000 claims description 5
- 125000000468 ketone group Chemical group 0.000 claims description 5
- FYGDTMLNYKFZSV-UHFFFAOYSA-N mannotriose Natural products OC1C(O)C(O)C(CO)OC1OC1C(CO)OC(OC2C(OC(O)C(O)C2O)CO)C(O)C1O FYGDTMLNYKFZSV-UHFFFAOYSA-N 0.000 claims description 5
- FYGDTMLNYKFZSV-BYLHFPJWSA-N β-1,4-galactotrioside Chemical compound O[C@@H]1[C@@H](O)[C@H](O)[C@@H](CO)O[C@H]1O[C@@H]1[C@H](CO)O[C@@H](O[C@@H]2[C@@H](O[C@@H](O)[C@H](O)[C@H]2O)CO)[C@H](O)[C@H]1O FYGDTMLNYKFZSV-BYLHFPJWSA-N 0.000 claims description 5
- JZNWSCPGTDBMEW-UHFFFAOYSA-N Glycerophosphorylethanolamin Natural products NCCOP(O)(=O)OCC(O)CO JZNWSCPGTDBMEW-UHFFFAOYSA-N 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 4
- 125000002791 glucosyl group Chemical group C1([C@H](O)[C@@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims description 4
- 230000003612 virological effect Effects 0.000 abstract description 56
- 229920001223 polyethylene glycol Polymers 0.000 description 67
- 108090000623 proteins and genes Proteins 0.000 description 60
- 239000002202 Polyethylene glycol Substances 0.000 description 57
- YMWUJEATGCHHMB-UHFFFAOYSA-N Dichloromethane Chemical compound ClCCl YMWUJEATGCHHMB-UHFFFAOYSA-N 0.000 description 50
- 239000002479 lipoplex Substances 0.000 description 45
- 210000004027 cell Anatomy 0.000 description 43
- 229910052739 hydrogen Inorganic materials 0.000 description 37
- 102000004169 proteins and genes Human genes 0.000 description 33
- 229910001868 water Inorganic materials 0.000 description 32
- OKKJLVBELUTLKV-UHFFFAOYSA-N Methanol Chemical compound OC OKKJLVBELUTLKV-UHFFFAOYSA-N 0.000 description 30
- 125000000217 alkyl group Chemical group 0.000 description 30
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 30
- 235000018102 proteins Nutrition 0.000 description 29
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 26
- 230000003993 interaction Effects 0.000 description 25
- 125000003729 nucleotide group Chemical group 0.000 description 25
- 108090000765 processed proteins & peptides Proteins 0.000 description 25
- 239000002773 nucleotide Substances 0.000 description 24
- 238000002347 injection Methods 0.000 description 20
- 239000007924 injection Substances 0.000 description 20
- 108020004999 messenger RNA Proteins 0.000 description 20
- 150000001412 amines Chemical class 0.000 description 19
- 239000000243 solution Substances 0.000 description 19
- 101150041968 CDC13 gene Proteins 0.000 description 18
- HEDRZPFGACZZDS-MICDWDOJSA-N Trichloro(2H)methane Chemical compound [2H]C(Cl)(Cl)Cl HEDRZPFGACZZDS-MICDWDOJSA-N 0.000 description 18
- 230000003828 downregulation Effects 0.000 description 18
- 230000000694 effects Effects 0.000 description 18
- 238000003786 synthesis reaction Methods 0.000 description 18
- 210000001519 tissue Anatomy 0.000 description 18
- 239000004215 Carbon black (E152) Substances 0.000 description 17
- 238000007792 addition Methods 0.000 description 17
- 150000001875 compounds Chemical class 0.000 description 17
- 229930195733 hydrocarbon Natural products 0.000 description 17
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 16
- 239000004471 Glycine Substances 0.000 description 16
- 230000015572 biosynthetic process Effects 0.000 description 16
- 150000002430 hydrocarbons Chemical class 0.000 description 16
- 210000004185 liver Anatomy 0.000 description 16
- 239000002904 solvent Substances 0.000 description 16
- 239000002253 acid Substances 0.000 description 15
- 125000000524 functional group Chemical group 0.000 description 15
- 150000003839 salts Chemical class 0.000 description 15
- 239000000126 substance Substances 0.000 description 15
- 238000003260 vortexing Methods 0.000 description 15
- 241001465754 Metazoa Species 0.000 description 14
- 238000006243 chemical reaction Methods 0.000 description 14
- 125000005842 heteroatom Chemical group 0.000 description 14
- 238000004128 high performance liquid chromatography Methods 0.000 description 14
- 238000001890 transfection Methods 0.000 description 14
- 229920000768 polyamine Polymers 0.000 description 13
- 229920005989 resin Polymers 0.000 description 13
- 239000011347 resin Substances 0.000 description 13
- 238000001644 13C nuclear magnetic resonance spectroscopy Methods 0.000 description 12
- VHYFNPMBLIVWCW-UHFFFAOYSA-N 4-Dimethylaminopyridine Chemical compound CN(C)C1=CC=NC=C1 VHYFNPMBLIVWCW-UHFFFAOYSA-N 0.000 description 12
- 206010061218 Inflammation Diseases 0.000 description 12
- ZMANZCXQSJIPKH-UHFFFAOYSA-N Triethylamine Chemical compound CCN(CC)CC ZMANZCXQSJIPKH-UHFFFAOYSA-N 0.000 description 12
- 241000700605 Viruses Species 0.000 description 12
- KRKNYBCHXYNGOX-UHFFFAOYSA-N citric acid Chemical compound OC(=O)CC(O)(C(O)=O)CC(O)=O KRKNYBCHXYNGOX-UHFFFAOYSA-N 0.000 description 12
- 238000011534 incubation Methods 0.000 description 12
- 230000004054 inflammatory process Effects 0.000 description 12
- 239000002245 particle Substances 0.000 description 12
- 241000282414 Homo sapiens Species 0.000 description 11
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- 230000002757 inflammatory effect Effects 0.000 description 11
- LDGWQMRUWMSZIU-LQDDAWAPSA-M 2,3-bis[(z)-octadec-9-enoxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCCOCC(C[N+](C)(C)C)OCCCCCCCC\C=C/CCCCCCCC LDGWQMRUWMSZIU-LQDDAWAPSA-M 0.000 description 10
- IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 description 10
- CSNNHWWHGAXBCP-UHFFFAOYSA-L Magnesium sulfate Chemical compound [Mg+2].[O-][S+2]([O-])([O-])[O-] CSNNHWWHGAXBCP-UHFFFAOYSA-L 0.000 description 10
- 241000699670 Mus sp. Species 0.000 description 10
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- 208000006454 hepatitis Diseases 0.000 description 10
- 150000007523 nucleic acids Chemical class 0.000 description 10
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 9
- RYHBNJHYFVUHQT-UHFFFAOYSA-N 1,4-Dioxane Chemical compound C1COCCO1 RYHBNJHYFVUHQT-UHFFFAOYSA-N 0.000 description 9
- WEVYAHXRMPXWCK-UHFFFAOYSA-N Acetonitrile Chemical compound CC#N WEVYAHXRMPXWCK-UHFFFAOYSA-N 0.000 description 9
- 108020004414 DNA Proteins 0.000 description 9
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- 229940079593 drug Drugs 0.000 description 9
- LEQAOMBKQFMDFZ-UHFFFAOYSA-N glyoxal Chemical compound O=CC=O LEQAOMBKQFMDFZ-UHFFFAOYSA-N 0.000 description 9
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- KHIWWQKSHDUIBK-UHFFFAOYSA-N periodic acid Chemical compound OI(=O)(=O)=O KHIWWQKSHDUIBK-UHFFFAOYSA-N 0.000 description 9
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- XEKOWRVHYACXOJ-UHFFFAOYSA-N Ethyl acetate Chemical compound CCOC(C)=O XEKOWRVHYACXOJ-UHFFFAOYSA-N 0.000 description 8
- PEDCQBHIVMGVHV-UHFFFAOYSA-N Glycerol Natural products OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 description 8
- NQRYJNQNLNOLGT-UHFFFAOYSA-N Piperidine Chemical compound C1CCNCC1 NQRYJNQNLNOLGT-UHFFFAOYSA-N 0.000 description 8
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- KSXTUUUQYQYKCR-LQDDAWAPSA-M 2,3-bis[[(z)-octadec-9-enoyl]oxy]propyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCC\C=C/CCCCCCCC(=O)OCC(C[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC KSXTUUUQYQYKCR-LQDDAWAPSA-M 0.000 description 6
- QBXODCKYUZNZCY-UHFFFAOYSA-N 2-[(2-methylpropan-2-yl)oxycarbonylamino]oxyacetic acid Chemical compound CC(C)(C)OC(=O)NOCC(O)=O QBXODCKYUZNZCY-UHFFFAOYSA-N 0.000 description 6
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Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2121/00—Preparations for use in therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
- Y10T—TECHNICAL SUBJECTS COVERED BY FORMER US CLASSIFICATION
- Y10T428/00—Stock material or miscellaneous articles
- Y10T428/29—Coated or structually defined flake, particle, cell, strand, strand portion, rod, filament, macroscopic fiber or mass thereof
- Y10T428/2982—Particulate matter [e.g., sphere, flake, etc.]
- Y10T428/2984—Microcapsule with fluid core [includes liposome]
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Organic Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Dispersion Chemistry (AREA)
- Physics & Mathematics (AREA)
- Manufacturing & Machinery (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La présente invention concerne un vecteur de livraison non viral comprenant un liposome, dans lequel un ou plusieurs lipides du liposome sont couplés, d~une manière réversible ou irréversible, à un ou plusieurs polymères et dans lequel le liposome comprend du siARN.
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
GB0418172.3 | 2004-08-13 | ||
GBGB0418172.3A GB0418172D0 (en) | 2004-08-13 | 2004-08-13 | Vector |
US61408504P | 2004-09-09 | 2004-09-09 | |
US60/614,085 | 2004-09-09 | ||
PCT/GB2005/002634 WO2006016097A2 (fr) | 2004-08-13 | 2005-07-06 | Vecteur |
Publications (1)
Publication Number | Publication Date |
---|---|
CA2576923A1 true CA2576923A1 (fr) | 2006-02-16 |
Family
ID=33017523
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CA002576923A Abandoned CA2576923A1 (fr) | 2004-08-13 | 2005-07-06 | Vecteur |
Country Status (8)
Country | Link |
---|---|
US (1) | US20080063701A1 (fr) |
EP (1) | EP1814594A2 (fr) |
CN (1) | CN101094691A (fr) |
AU (1) | AU2005271100A1 (fr) |
CA (1) | CA2576923A1 (fr) |
GB (1) | GB0418172D0 (fr) |
WO (1) | WO2006016097A2 (fr) |
ZA (1) | ZA200701246B (fr) |
Families Citing this family (30)
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BRPI0713331A2 (pt) * | 2006-06-30 | 2012-03-13 | Hokkaido System Science Co., Ltd. | composição para a transfecção de ácido nucléico |
EP2125031B1 (fr) | 2006-12-19 | 2017-11-01 | Marina Biotech, Inc. | Lipides et ensembles lipidiques comprenant des éléments d'amélioration de la transfection |
AR067997A1 (es) * | 2007-08-24 | 2009-10-28 | Novartis Ag | Compuestos organicos |
GB2458473A (en) | 2008-03-17 | 2009-09-23 | Imuthes Ltd | 3'-O-allyl- and 3'-O-carboxymethyl- 2'-aminosaccharide derivatives, & amides thereof with peptides, as adjuvants |
WO2009124137A2 (fr) * | 2008-04-01 | 2009-10-08 | Mount Sinai School Of Medicine Of New York University | Procédé de suppression d’une transcription de gène par méthylation d’histone lysine |
CN102112110A (zh) * | 2008-06-06 | 2011-06-29 | 米尔纳医疗股份有限公司 | 用于RNAi试剂体内递送的新型组合物 |
EP2184054A1 (fr) | 2008-11-08 | 2010-05-12 | Lipoxen Technologies Limited | Livraison ARN à faible interférence |
AU2010210593B2 (en) * | 2009-02-04 | 2015-09-03 | The Brigham And Women's Hospital, Inc. | Nanoscale platinum compounds and methods of use thereof |
PT3338765T (pt) | 2009-12-01 | 2019-03-18 | Translate Bio Inc | Derivado de esteróide adequado para a administração de arnm em doenças genéticas humanas |
SG10201407996PA (en) | 2009-12-23 | 2015-01-29 | Novartis Ag | Lipids, lipid compositions, and methods of using them |
US9028797B2 (en) | 2010-02-26 | 2015-05-12 | Nagasaki University | Composite body for antigen or drug delivery |
AU2012267531B2 (en) | 2011-06-08 | 2017-06-22 | Translate Bio, Inc. | Lipid nanoparticle compositions and methods for mRNA delivery |
US20140161876A1 (en) * | 2011-07-15 | 2014-06-12 | Konica Minolta, Inc. | Liposome-containing preparation utilizing dissolution aid, and method for producing same |
CN102973506B (zh) * | 2011-09-05 | 2015-06-03 | 中国科学院深圳先进技术研究院 | 阳离子脂质体及其制备方法 |
GB201116248D0 (en) * | 2011-09-20 | 2011-11-02 | Glaxosmithkline Biolog Sa | Liposome production using isopropanol |
EP3536787A1 (fr) | 2012-06-08 | 2019-09-11 | Translate Bio, Inc. | Polynucléotides résistant aux nucléases et leurs utilisations |
EA201492055A1 (ru) | 2012-06-08 | 2015-11-30 | Шир Хьюман Дженетик Терапис, Инк. | ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ |
JP6586075B2 (ja) | 2013-03-14 | 2019-10-02 | トランスレイト バイオ, インコーポレイテッド | メッセンジャーrnaの精製方法 |
HUE042640T2 (hu) | 2013-03-14 | 2019-07-29 | Translate Bio Inc | CFTR-mRNS-készítmények, valamint kapcsolódó eljárások és alkalmazások |
EP3060258A1 (fr) | 2013-10-22 | 2016-08-31 | Shire Human Genetic Therapies, Inc. | Thérapie à l'arnm pour la phénylcétonurie |
ES2707966T3 (es) | 2013-10-22 | 2019-04-08 | Translate Bio Inc | Terapia de ARNm para la deficiencia en síntesis de argininosuccinato |
KR20220158867A (ko) | 2014-04-25 | 2022-12-01 | 샤이어 휴먼 지네틱 테라피즈 인크. | 메신저 rna 의 정제 방법 |
EP3169309B1 (fr) | 2014-07-16 | 2023-05-10 | Novartis AG | Procédé d'encapsulation d'un acide nucléique dans une nanoparticule lipidique hôte |
CN105985386B (zh) | 2015-02-11 | 2018-06-26 | 大连民族学院 | 一类蔗糖酯型阳离子基因载体及其制备方法 |
CZ307452B6 (cs) * | 2016-11-03 | 2018-09-05 | Vysoká škola chemicko-technologická v Praze | Aminooxylipidy pro konstrukci samoskladných liposomálních systémů umožňujících jejich následnou modifikaci biologicky funkčními molekulami |
EP3585417B1 (fr) | 2017-02-27 | 2023-02-22 | Translate Bio, Inc. | Méthode de préparation d'arnm cftr à codons optimisés |
WO2018213476A1 (fr) | 2017-05-16 | 2018-11-22 | Translate Bio, Inc. | Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr |
CA3108544A1 (fr) | 2018-08-24 | 2020-02-27 | Translate Bio, Inc. | Procedes de purification d'arn messager |
CN113116819B (zh) * | 2021-04-24 | 2022-09-20 | 郑州大学 | 一种载siRNA纳米脂质杂化胶束及其制备方法和应用 |
CN114249791A (zh) * | 2021-12-27 | 2022-03-29 | 北京工商大学 | 一种甾醇衍生的酰胺基寡肽型表面活性剂及其制备方法 |
Family Cites Families (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2911197A (en) * | 1996-05-24 | 1998-01-05 | Imperial College Of Science, Technology And Medicine | Polycationic sterol derivatives as transfection agents |
US6120751A (en) * | 1997-03-21 | 2000-09-19 | Imarx Pharmaceutical Corp. | Charged lipids and uses for the same |
GB9930533D0 (en) * | 1999-12-23 | 2000-02-16 | Mitsubishi Tokyo Pharm Inc | Nucleic acid delivery |
US20020188023A1 (en) * | 2000-12-12 | 2002-12-12 | Michael Jorgensen | Compound |
US6921812B1 (en) * | 2001-07-03 | 2005-07-26 | Isis Pharmaceuticals, Inc. | Methods of modulating pharmacokinetics of oligonucleotides |
AU2003237249A1 (en) * | 2002-05-24 | 2003-12-12 | Isis Pharmaceuticals, Inc. | Oligonucleotides having modified nucleoside units |
US7148342B2 (en) * | 2002-07-24 | 2006-12-12 | The Trustees Of The University Of Pennyslvania | Compositions and methods for sirna inhibition of angiogenesis |
US20050002999A1 (en) * | 2003-06-04 | 2005-01-06 | Rahul Mehta | Long-circulating liposomal compositions |
EP2567693B1 (fr) * | 2003-07-16 | 2015-10-21 | Protiva Biotherapeutics Inc. | ARN interférents encapsulés dans des lipides |
JP4842821B2 (ja) * | 2003-09-15 | 2011-12-21 | プロチバ バイオセラピューティクス インコーポレイティッド | ポリエチレングリコール修飾脂質化合物およびその使用 |
WO2005121348A1 (fr) * | 2004-06-07 | 2005-12-22 | Protiva Biotherapeutics, Inc. | Arn interferant encapsule dans des lipides |
-
2004
- 2004-08-13 GB GBGB0418172.3A patent/GB0418172D0/en not_active Ceased
-
2005
- 2005-07-01 US US11/172,641 patent/US20080063701A1/en not_active Abandoned
- 2005-07-06 CA CA002576923A patent/CA2576923A1/fr not_active Abandoned
- 2005-07-06 ZA ZA200701246A patent/ZA200701246B/xx unknown
- 2005-07-06 WO PCT/GB2005/002634 patent/WO2006016097A2/fr active Application Filing
- 2005-07-06 EP EP05757661A patent/EP1814594A2/fr not_active Withdrawn
- 2005-07-06 AU AU2005271100A patent/AU2005271100A1/en not_active Abandoned
- 2005-07-06 CN CNA2005800339537A patent/CN101094691A/zh active Pending
Also Published As
Publication number | Publication date |
---|---|
ZA200701246B (en) | 2008-12-31 |
GB0418172D0 (en) | 2004-09-15 |
AU2005271100A1 (en) | 2006-02-16 |
US20080063701A1 (en) | 2008-03-13 |
WO2006016097A2 (fr) | 2006-02-16 |
CN101094691A (zh) | 2007-12-26 |
WO2006016097A3 (fr) | 2006-11-23 |
EP1814594A2 (fr) | 2007-08-08 |
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